PTC Therapeutics, Inc. PTCT
PTC Therapeutics, Inc. (Nasdaq: PTCT) is a rare genetic drug developer. Its lead program, ST-920 (isaralgagene civaparvovec), is in Phase 3 for Fabry disease. Its next catalyst on record is ST-920: BLA submission completion (Q4 2026).
Upcoming catalysts
No catalyst is dated to the next 30 days or to an FDA action date.
Next further out: ST-920 — BLA submission completion (Q4 2026). 4 catalysts on record beyond the next 30 days; subscribers see each with its filing sentence.
Pipeline
| Program | Indication | Modality | Stage |
|---|---|---|---|
| Sephience (sepiapterin) | PKU | small molecule | Approved |
| Emflaza (deflazacort) | DMD | small molecule | Approved |
| Upstaza/Kebilidi (eladocagene exuparvovec) | AADC deficiency | gene therapy | Approved |
| Translarna (ataluren) | nmDMD | small molecule | Approved |
| ST-920 (isaralgagene civaparvovec) | Fabry disease | gene therapy | Phase 3 |
| votoplam | Huntington's disease | small molecule | Phase 3 |
| vatiquinone | Friedreich's ataxia | small molecule | Phase 3 |
| PTC844 | T-cell and B-cell immunity (specific indication unclear) | small molecule | Phase 2 |
| PTC612 | unclear (obesity, cardiovascular disease mentioned) | small molecule | Phase 1 |
Recent results and decisions
- Oct 29, 2026submitted PTC expects to complete BLA submission for ST-920 Fabry disease gene therapy in Q4 2026.
ST-920 Fabry disease BLA submission expected to be completed in Q4 2026
Filing - Oct 29, 2026started PTC initiated a Phase 2a study of PTC844, a next-generation DHODH inhibitor.
Initiated Phase 2a study of PTC844, a next-generation DHODH inhibitor
Filing - Oct 29, 2026submitted ST-920 Fabry disease gene therapy BLA submission expected to be completed in Q4 2026.
ST-920 Fabry disease BLA submission expected to be completed in Q4 2026
Filing
7 more on record.
For subscribers
- Cash runway against each catalyst, with the company's own runway statement
- Track record: every Phase 3 readout, FDA decision and guidance slip since 2014
- Expected move for each dated catalyst, from comparable past events
- Phase 2 evidence behind each Phase 3 readout
- Companies on the same target or class
- Full catalyst list with filing sentences, deals, registered trials, Excel export
Other phase 3 rare genetic companies
The free Monday brief
Last week's readouts and FDA decisions and the dated catalysts in the week ahead, each with its filing sentence.